Prednisone and deflazacort in Duchenne muscular dystrophy: a patient perspective and plain language summary publication of the Cincinnati study
This plain language summary of publication, co-authored by a patient, a caregiver and a treating physician, includes the patient perspective and help patients with Duchenne muscular dystrophy and their caregivers understand the results of the Cincinnati study.
Plain language summary of the long-term effect of ataluren in patients with Duchenne muscular dystrophy
Read the latest plain language summary looking at how ataluren treatment in combination with standard of care can delay important signs of disease progression in patients with Duchenne muscular dystrophy.
Read the article summarising the results of two studies looking at vosoritide as a potential treatment for children with achondroplasia.
The first of it’s kind published in a peer-reviewed journal, this article published in Future Oncology provides a lay language summary of the ENLIVEN study.